Biogen’s Qalsody (tofersen) has been recommended by the European Medicines Agency’s human medicines committee to treat amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.
If approved by the European Commission, which is expected to make its decision in the second quarter of this year, Qalsody will become the first treatment authorised in the EU to target a genetic cause of ALS.
ALS is a rare and progressive neurodegenerative disease resulting in the loss of motor neurons in the brain and the spinal cord that are responsible for controlling voluntary muscle movement.
Multiple genes have been implicated in ALS and mutations in the SOD1 gene are responsible for approximately 2% of all cases of the disease.
Qalsody, which Biogen has licenced from Ionis Pharmaceuticals, is an antisense oligonucleotide designed to bind to SOD1 mRNA and reduce SOD1 protein production.
The Committee for Medicinal Products for Human Use’s (CHMP) positive opinion on the drug was supported by data from the 28-week phase 3 VALOR study, in which patients treated with Qalsody experienced reductions of 60% in plasma neurofilament light chain – a marker of neurodegeneration – compared to the placebo cohort.
Trends towards improvement in the physical abilities of Qalsody-treated patients were also seen compared to those who received placebo.
Priya Singhal, head of development at Biogen, said: “The CHMP’s positive opinion reinforces the impact Qalsody can have in SOD1-ALS and further demonstrates Biogen’s commitment to address the unmet needs of people living with ALS and neuromuscular diseases.”
Qalsody was granted accelerated approval by the US Food and Drug Administration in April of last year for the same patient population.
“The CHMP’s recommendation in support of Qalsody approval provides new hope for the ALS community in Europe,” said Philip Van Damme, professor of neurology and director of the Neuromuscular Reference Center at the University Hospital Leuven in Belgium.
“This is a significant milestone for the entire ALS community – for the first time we have a treatment that led to sustained reductions in neurofilament, a marker of axonal injury and neurodegeneration,” he added.